Clinical trials: Skeletal Disorder
Ischemic Heart Failure, Ischemic Heart Disease
Study to Examine the Safety and Effectiveness of Implanted Skeletal Muscle Cells (Cells Removed From the Thigh Muscle) Into Scarred Areas of Heart Muscle After Heart Attack.
Study to examine the safety and effectiveness of implanted skeletal muscle cells (cells removed from the thigh muscle) into scarred areas of heart muscle after heart attack.
- Conditions
- Ischemic Heart Failure, Ischemic Heart Disease, Ischemic Cardiomyopathy
- Trial name
- NCT00102128
- Testing
- Cellular Therapy (Cultured Autologous Skeletal Myoblast Transplantation)
- Where
- 28 sites in Belgium, France and 4 more
- Sponsor
- Genzyme, a Sanofi Company
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.
- Trial name
- VITALITY-ALS (NCT02496767)
- Testing
- Tirasemtiv
- Where
- 81 sites in United States, Belgium and 9 more
- Volunteers
- 744 people
- Sponsor
- Cytokinetics
Thalassemia
Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events
The evaluation of efficacy is performed by laboratory monitoring of bone density and resorption markers and clinical monitoring of bone density improvement.
- Trial name
- NCT00346242
- Testing
- Zoledronic Acid
- Where
- Greece
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Multiple Myeloma
Zoledronic Acid Treatment (Every 4 or 12 Weeks) to Prevent Skeletal Complications in Advanced Multiple Myeloma Participants
This study evaluated the effectiveness and safety of a dosing method for zoledronic acid in preventing skeletal complications in multiple myeloma participants who have been on an intravenous (IV) bisphosphonate for about one to…
- Trial name
- Z-MARK (NCT00622505)
- Testing
- zoledronic acid
- Where
- 42 sites in United States
- Volunteers
- 121 people
- Sponsor
- Novartis Pharmaceuticals
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Sarcopenia, Body Composition
Validation of a Body-Composition Segmentation Software on a Diverse Public CT Scan Cohort
This study evaluates the standalone performance of Soma, a deep-learning software developed by Nucleo Research, Inc. for the automated segmentation of body-composition tissues (skeletal muscle, subcutaneous adipose tissue…
- Conditions
- Sarcopenia, Body Composition, Obesity
- Trial name
- SOMA (NCT07600866)
- Testing
- Soma Body-Composition Segmentation Software
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Nucleo Research, Inc.
Multiple Myeloma
Study Comparing STR (Skeletal Targeted Radiotherapy) Plus Melphalan to Melphalan Alone, With Stem Cell Transplant in Multiple Myeloma
STR (Skeletal Targeted Radiotherapy, 166Ho-DOTMP) is an investigational radiopharmaceutical that delivers radiation directly to cancer cells in the bone and bone marrow.
- Trial name
- NCT00083564
- Testing
- STR(TM) (Skeletal Targeted Radiotherapy, Holmium-166-DOTMP)
- Where
- 21 sites in United States, Canada
- Volunteers
- 240 people
- Sponsor
- Poniard Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Multiple Myeloma Bone Disease
A Study to Assess BHQ880 in Combination With Zoledronic Acid in Relapsed or Refractory Myeloma Patients
This study has two portions, a phase I portion and a phase II portion. The purpose of the phase I portion is to assess the maximum-tolerated dose (MTD) and to characterize dose limiting toxicity (DLT) of escalating doses of…
- Trial name
- NCT00741377
- Testing
- BHQ880, Zoledronic acid
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- BioMarin Pharmaceutical
Post-Acute Sequelae of SARS-CoV-2 (PASC) Infection
Efficacy, Safety, Tolerability of AXA1125 in Fatigue After COVID-19 Infection
This study will compare the effects of AXA1125, an orally active mixture of amino acids, compared to placebo, on improving muscle function (metabolism) following moderate exercise in subjects with fatigue-Predominant Post-Acute…
- Trial name
- NCT05152849
- Testing
- AXA1125
- Where
- United Kingdom
- Volunteers
- 41 people
- Sponsor
- Axcella Health, Inc
Prostate Cancer, Neoplasm Metastasis
BAY88-8223, Dose Finding Study in Patients With HRPC
The purpose of this study is to evaluate the effectiveness of the investigational radioisotope Radium-223, Alpharadin, in treatment of men with prostate cancer and bone metastases that no longer respond to hormonal treatment.
- Trial name
- NCT00337155
- Testing
- Radium-223 dichloride (BAY88-8223)
- Where
- United Kingdom
- Volunteers
- 122 people
- Sponsor
- Bayer
Prostate Cancer, Metastases
Alpharadin™ (Radium-223 Chloride) Safety and Dosimetry With HRPC That Has Metastasized to the Skeleton
The purpose of the study is to investigate the safety, biodistribution, radiation dosimetry and pharmacokinetics of three intravenous escalating dose levels of Xofigo (Alpharadin).
- Conditions
- Prostate Cancer, Metastases, Pharmacokinetics
- Trial name
- NCT00748046
- Testing
- Radium-223 chloride (BAY88-8223)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Bayer
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Obesity & Overweight
Effect of a Food for Special Medical Purposes on Muscle Mass Preservation During GLP-1 RAs Weight Loss Treatment
This study evaluates whether a Food for Special Medical Purposes (FSMP) can help to preserve appendicular skeletal muscle mass (ASMM) in adults undergoing weight loss treatment with GLP-1 receptor agonists or dual GIP/GLP-1…
- Trial name
- MYO-PRESERVE (NCT07393360)
- Testing
- Myosave®
- Where
- 3 sites in Italy
- Volunteers
- 144 people
- Sponsor
- Pharmanutra S.p.a.
Fibrodysplasia Ossificans Progressiva (FOP)
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Primary Objective(s): To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) To describe the prevalence of clinical characteristics of…
- Trial name
- NCT06064656
- Testing
- Non-Interventional
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Regeneron Pharmaceuticals
Diabetes Mellitus, Type 2
Effect of Vildagliptin on Fat and Muscle Metabolism in Patients With Type 2 Diabetes
Please note this study is not being conducted in the United States. The purpose of this study is to test the hypothesis that acute DPP-4 inhibition with vildagliptin improves fat and muscle metabolism in patients with type 2…
- Trial name
- NCT00380445
- Testing
- Vildagliptin
- Where
- 2 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Novartis
Developmental Delay, Skeletal Abnormalities
Biomarker for Sly Disease (MPS VII) (BioSly)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Sly disease from blood (plasma)
- Conditions
- Developmental Delay, Skeletal Abnormalities, Hepatomegaly, Splenomegaly
- Trial name
- BioSly (NCT02298699)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00365131
- Testing
- Cerezyme (imiglucerase for injection)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Genzyme, a Sanofi Company